PASADENA,
Calif. — February 26, 2013 — Arrowhead Research Corporation (NASDAQ:
ARWR), a targeted therapeutics company, today announced the publication
of data demonstrating multi-log reductions in hepatitis B viral DNA and
proteins lasting over 30 days after a single injection in animal models.
This suggests that Arrowhead’s RNAi-based candidate ARC-520 has the
potential to treat chronic hepatitis B virus infection in a
fundamentally different manner, with the goal of achieving a functional
cure. The paper, entitled “Hepatocyte-targeted RNAi therapeutics for the
treatment of chronic hepatitis B virus infection,” by Wooddell et al,
was published online ahead of print in the journal Molecular Therapy(doi:10.1038/mt.2013.31).

In the publication, Arrowhead scientists describe the use of a novel
Dynamic PolyConjugate (DPC) technology to deliver small interfering RNAs
(siRNAs) designed against the hepatitis B virus (HBV). This DPC
technology incorporates a biodegradable peptide composed of naturally
occurring amino acids and a liver-targeted molecule that is co-injected
with a cholesterol-conjugated siRNA (chol-siRNA). In
proof-of-concept studies, utilization of this DPC to deliver chol-siRNA
targeting Factor 7 to non-human primates results in >99% knockdown
of target gene expression and >80% knockdown for over one month after
a single injection. Multi-dose studies in mice showed no diminution of
knockdown activity or toxicity upon repeated injection at therapeutic
doses. In transient and transgenic mouse models of HBV infection, a
single co-injection of DPC with chol-siRNA targeting HBV sequences
resulted in multi-log knockdown of HBV RNA, proteins and viral DNA with
long duration of effect.

“This publication is important because it speaks to a specific
product and a broader platform,” said Dr. Christopher Anzalone,
President and Chief Executive Officer. “These data suggest that ARC-520
could be a powerful therapy for chronic HBV infection, a disease with
350 million infected people worldwide and no cure. We are on schedule to
file with regulatory authorities next quarter to begin first-in-human
studies. During phase 1 we will be able to measure the drug’s ability to
knock down production of new infectious virus as well as viral
proteins, including s-antigen, e-antigen, and the core protein that
forms the capsid. The ability to substantially knock down these viral
proteins is what is unique about ARC-520 and what many in the field
believe will be necessary to revive the host immune response and
potentially provide a functional cure, which no other current therapy
can reliably do. More broadly, this paper reports on a delivery system
capable of extremely efficient gene silencing that can be used for a
variety disease targets.”

About Arrowhead Research Corporation

Arrowhead Research Corporation is a clinical stage targeted
therapeutics company with development programs in oncology, obesity, and
chronic hepatitis B virus infection. The company is leveraging its
platform technologies to design and develop peptide-drug conjugates
(PDCs) that specifically home to cell types of interest while sparing
off-target tissues, create targeted drugs based on the gene silencing
RNA interference (RNAi) mechanism, and work with partners to create
improved versions of traditional small molecule drugs.

Paul J. Pockros: “Most believe that nucleoside polymerase inhibitors [NPI] will be the backbone of antiviral therapies for HCV,” researcher Paul J. Pockros, MD, director of the Liver Disease Center and the SC Liver Research Consortium at Scripps Clinic in La Jolla, Calif., told Healio.com. “The first NPI was R1626 (balapiravir), [which] proved to be potent, but toxic. Therefore, the critical need was to develop a safe NPI.”

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